Is there like a check list available for the processes left to get this moving along in the USA? I feel like the urgency just isnt there. How close is this to even happening? Ive asked months ago how close the funding was (or how far off it is).

10-26-2013, 02:39 PM

Wise Young

Just because I don't post about this every day doesn't mean that people are not working very hard on this. It takes a lot of work to set up global clinical trials, to raise the money for the trials, and to write and publish the papers in the best journals. We closed the database at the end of July and have been working hard to verify the data so that the analysis can start. We have raised most of the funds for the trial in China. In the U.S., we have been working very hard helping Stemcyte get an IPO so that they can support the trial and they recently received approval for an IPO in Taiwan. We are transferring the cell processing technology to China so that that we can submit the IND (application for clinical trial) to the Ministry of Health in China. We will be submitting INDs to the FDA and the EMA (the regulatory agency for Europe) in December or January. In the meantime, of course, we have been working on three papers to be submitted to various journals, i.e. the papers describing how the cells were processed, the phase I trial in Hong Kong with the MR-DTI studies, the phase II trial in Kunming that assessed walking recovery in 20 subjects with chronic complete spinal cord injuries. We are doing all of this with a tiny team of people and very little funding. So, please be patient. Even companies spending hundreds of millions are not doing this as fast as we are. Wise.

10-26-2013, 06:56 PM

#LHB#

So why is the U.S. sucking hind tit? Have we in the U.S. not raised enough money for the trial or is there another reason.

10-26-2013, 07:10 PM

Barrington314mx

Thanks for the reply Doc. Why does it seem that its much easier to get trials approved and raise money in China? I did not know you planned to submit to FDA within a few months. Thats good to know. Do you have an idea of how long it takes the FDA to approve?

10-27-2013, 07:08 PM

Wise Young

Quote:

Originally Posted by Barrington314mx

Thanks for the reply Doc. Why does it seem that its much easier to get trials approved and raise money in China? I did not know you planned to submit to FDA within a few months. Thats good to know. Do you have an idea of how long it takes the FDA to approve?

What can I say? Some of our representatives in Congress seem determined to transform the United States into a third world country. For the past decade, Congress has flat-lined funding of biomedical research, even though everybody knows and Congress certainly should know that the trillion dollar U.S. pharmaceutical industry depends on NIH funded research for most of its advances. The United States is the last "developed" nation in the world to provide universal health care for its citizen with so-called "Obamacare". Yet, we just had a 3-week shutdown of the government over this issue. There is little or no governmental funding of clinical trials, except perhaps for cancer, in the U.S. I believe that the U.S. is the most expensive country in the world to do clinical trials. The U.S. is falling behind the world in stem cells, spinal cord injury, biomedical research, and clinical trials. Congress seems to think that this is okay.

China decided two years ago that they are going to devote 0.5% of its $7 trillion GDP to translational science and medicine. They are now investing 3.5% of their GDP every year into their universities. The additional 0.5% is about $35 billion, more than all of NIH. In other words, they are serious about transforming their basic sciences to yield products that benefit their people. ChinaSCINet will be getting funding from the central government, provincial government, and also from charitable donations in Hong Kong. China is sending their best and brightest to the United States to learn how to do research, go back to China, and laugh all the way to the bank. Perchance, have you noticed that the authors of almost all the major papers in spinal cord injury research are now Chinese? This is no accident. They are now publishing more papers on spinal cord injury research from China than the U.S.

By law, the U.S. FDA must respond to an IND (application for clinical trial) in 90 days with an approval or a reason why the application is not approved. If we have a good application, we may get it in the first round although that is very unlikely. If they want all sorts of additional animal data for safety, this may take a while. However, since we have shown safety in human studies, it is a matter of convincing them that this is the case and hope that they will approve the IND in the U.S. We shall see. It is important to get a really good application in from the get-go. This will make the next applications easier. More important, for the future, we must do a really good job with the clinical trials. All the i's must be dotted and the t's crossed.

We are just using manual needle insertion (without a micro manipulator) and an external micro injector. We have chosen specifically to avoid using the kind of device that the doctors in the NeuralStem trials are using and apparently has patented. We did so because the spinal cord pulsates (1-2 mm) every time there is respiration. If the needle is held by a micromanipulator that is rigidly fixed to something outside the body, the needle is being pushed and down in the spinal cord every few seconds. We therefore prefer the floating needle approach. The needle is manually inserted to 3-mm depth and then light held so that it moves with the tissue. We believe that this is faster, safer, and cheaper. Wise.

11-02-2013, 03:07 PM

Sue Pendleton

Ok. This just looked very close to what you were showing. I thought it did move with any movement of the patient. It seems there are more of these kind of delivery systems being patented for the brain and spinal cord to make injections and micro surgeries safer.

The question is how to apply this treatment (which is a genetic knockout of Eph4A) and who will pay for manufacturing the treatment, showing that it is safe, and then testing it in clinical trial. In my opinion, this is very far from clinical trial. It is not a treatment but a genetic condition that they tested. Somebody will have to replicate it and then figure out a way to silence Eph4A, show that it is safe to do so, test it in animals to find out the optimal dose, timing, and duration of therapy for restoring function, and then start doing Phase I, II, and III clinical trials. What they are reporting is just the first stage in the process. If they don't have some deep-pocketed backers, the treatment turns out to be dangerous in any way, and the treatment is not as effective as expected, the whole treatment may bite the dust.

For this reason, I have decided to establish a $100 million fund to take the most promising spinal cord injury therapies from discovery to market. We are now close to our first phase III trials. If the phase III trials successfully demonstrates that umbilical cord blood mononuclear cells and lithium improves locomotor recovery, I want to use the success to raise a significant amount of money that would be used to take therapies from bench to bedside. Of course, now it is the first (and the most difficult) time to prove that we have a therapy. We have to overcome all sorts of naysayers and skeptics. But, we should be able to set up such a fund that would continually be funded from successful trials. We should not have to re-invent the wheel every time we have to take a treatment to trial.

Wise.

11-09-2013, 12:35 AM

JamesMcM

#2031 Today, 12:35 AM Wise, how long will the phase 3 trial be following patient one, two years or more? And if UMCB cell and lithium are looking effective, do you think you China SCI.net will try to start a phase 1-2 safety trial for a second generation treatment as you stated above, Before all data is analyzed and published for the phase 3 Trial. Does you know if acorda has a patent for rights on Chase?